Farkas Henriette, Balla Zsuzsanna
Hungarian Angioedema Center of Reference and Excellence, Department of Internal Medicine and Haematology, Semmelweis University, Budapest, Hungary.
HNO-Praxis Schaffhausen, Schaffhausen, Switzerland.
Expert Opin Drug Saf. 2023 Jul-Dec;22(7):549-561. doi: 10.1080/14740338.2023.2226861. Epub 2023 Jun 21.
Hereditary angioedema (HAE) is characterized by recurrent subcutaneously and/or submucosally localized edematous swellings. The first symptoms often appear in childhood, and they may become more frequent and severe in puberty. Since the appearance of HAE attacks is unpredictable regarding the localization and the frequency, the attacks put a significant burden on the patients and crucially impacts their quality of life.
This review article analyzes the safety data acquired from the clinical trials conducted with the currently available medicinal products for the prophylactic treatment of hereditary angioedema due to C1 inhibitor deficiency and the safety data of observatory studies based on clinical practice. A review of the published literature was conducted using the PubMed database, clinical trials from ClinicalTrials.gov, and abstracts published at scientific conferences.
The currently available therapeutic products have a good safety and efficiency profile and the international guidelines recommend them as first-line treatments. The choice should be made based on the evaluation of the availability and the preference of the patient.
遗传性血管性水肿（HAE）的特征是反复出现皮下和/或粘膜下局限性水肿肿胀。最初症状常出现在儿童期，在青春期可能会变得更加频繁和严重。由于HAE发作的部位和频率不可预测，这些发作给患者带来了巨大负担，并对他们的生活质量产生了至关重要的影响。
本文综述分析了从目前用于因C1抑制剂缺乏导致的遗传性血管性水肿预防性治疗的药品所进行的临床试验中获得的安全性数据，以及基于临床实践的观察性研究的安全性数据。使用PubMed数据库、ClinicalTrials.gov上的临床试验以及在科学会议上发表的摘要对已发表的文献进行了综述。
目前可用的治疗产品具有良好的安全性和有效性，国际指南推荐它们作为一线治疗方法。应根据对可用性的评估和患者的偏好来做出选择。
